Outcome of hematopoietic stem cell transplantation (HCT) from HLA-matched related donor for Fanconi anemia (FA) in adolescents and adults: a retrospective study by Eastern Mediterranean Blood and Marrow Transplantation Group (EMBMT)
Hematopoietic Stem Cell Transplantation (HSCT) is the only potentially curative treatment option for the hematologic complications that occur in patients with Fanconi anemia (FA). In this study, we present a retrospective multicenter analysis from the Eastern Mediterranean Blood and Marrow Transplan...
Gespeichert in:
| Veröffentlicht in: | Bone marrow transplantation (Basingstoke) 2020-07, Vol.55 (7), p.1485-1490 |
|---|---|
| Hauptverfasser: | , , , , , , , , , , , , , , , , , , , , , , , , , |
| Format: | Artikel |
| Sprache: | eng |
| Schlagworte: | |
| Online-Zugang: | Volltext |
| Tags: |
Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!
|
| container_end_page | 1490 |
|---|---|
| container_issue | 7 |
| container_start_page | 1485 |
| container_title | Bone marrow transplantation (Basingstoke) |
| container_volume | 55 |
| creator | AlDawsari, Ghuzayel Elhaddad, Alaa El Fakih, Riad Ben Othman, Tarek Ahmed, Parvez Ghavamzadeh, Ardeshir Bazarbachi, Ali Dasouki, Majed J. Fathy, Gamal Alzahrani, Hazzaa Samra, Mohamed Torjemane, Lamia Satti, Tariq Mahmood Shaheen, Marwan Alfraih, Feras Ayas, Mouhab Alahmari, Ali Alhayli, Saud Nassar, Amr Abboud, Miguel Abdelfattah, Raafat El Solh, Hassan Hashmi, Shahrukh Elhassan, Tusneem Ahmed, Syed Osman Aljurf, Mahmoud |
| description | Hematopoietic Stem Cell Transplantation (HSCT) is the only potentially curative treatment option for the hematologic complications that occur in patients with Fanconi anemia (FA). In this study, we present a retrospective multicenter analysis from the Eastern Mediterranean Blood and Marrow Transplantation Group (EMBMT) of matched related donor HSCT for FA in adolescents and adults transplanted between 1988 and 2015. Forty-five patients received HSCT with a median age at transplant of 18 years, the interquartile range (IQR) (15–23.5); 25 (55.6%) patients were females and 20 (44.4%) were males. Conditioning regimen was fludarabine-based in 29 (64.4%) patients, irradiation-based in five (11.1%) patients, and the remaining patients received other combinations. Indication for HSCT was bone marrow failure in 39 (86.7%) and myelodysplastic syndrome in six (13.3%) patients. Stem cell source was bone marrow in 22 (48.9%), peripheral blood in 20 (44.4%), umbilical cord blood in one (2.2%), and combination of bone marrow and cord blood in two (4.4%) patients. Twenty-seven (60%) patients engrafted and five (11.1%) had primary engraftment failure. The median time to neutrophil engraftment was 14 days (range 10–21 days); median time for platelet engraftment was 17 days (10–33 days). The probability of developing grade II–IV acute GVHD for all patients was 7.0% and chronic GVHD 36.6%. No new malignancies were reported. The OS probability was 53.6% (95% CI, 38.3–68.9%) with a median follow-up of 13 months (95% CI, 1–240). Our HLA-matched related HSCT results in AYA patients with FA compare favorably with other reported international registry data. |
| doi_str_mv | 10.1038/s41409-020-0809-5 |
| format | Article |
| fullrecord | <record><control><sourceid>gale_proqu</sourceid><recordid>TN_cdi_proquest_miscellaneous_2352051507</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><galeid>A628316920</galeid><sourcerecordid>A628316920</sourcerecordid><originalsourceid>FETCH-LOGICAL-c474t-669ec9198f5828c7ab494e27b70bf74b38e401e70170a053ddaeda3ab05683953</originalsourceid><addsrcrecordid>eNp9ks2O0zAUhSMEYsrAA7BBlpBQuwj4N3Fm16naKVKr2ZS15SQ304wcO9gOaN6Yx8ClA0gjwcK6tu53j46vTpa9JfgjwUx-CpxwXOWY4hzLdBHPshnhZZELVojn2QzTQuaMFdVF9iqEe4wJ51i8zC4YxZRXUs6yH7dTbNwAyHXoCIOObnQ9xL5BIcKAGjAGRa9tGI22UcfeWTTfrg4L1Hk3oO1umaeh5ggt8mB0TLV11nnUpbPRtnG2R9rC0Gs03ywXqLdIt85AaMDGkFptek8mhiukk0T0LozQxP4bJAdT-4DqB7TWyYy3aA9tny7JDmiLro1z7S-BvfbefUeHJz5vvJtGNF_vr_eHxevsRadNgDeP9TL7slkfVtt8d3vzebXc5Q0vecyLooKmIpXshKSyKXXNKw60rEtcdyWvmQSOCZSYlFhjwdpWQ6uZrrEoJKsEu8zmZ93Ru68ThKiGPpzWmEy7KSjKBMWCCFwm9P0T9N5N3iZ3ivJSYE4EKf5PkSqJYSkT9eFM3WkD6gjaxGNwZjotIqhlQSUjRYITSM5gkzYdPHRq9P2g_YMiWJ1Spc6pUilV6pQqdfrTu0cLUz1A-2fid4wSQM9ASC17B_6vx3-r_gTOXdgf</addsrcrecordid><sourcetype>Aggregation Database</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>2419205088</pqid></control><display><type>article</type><title>Outcome of hematopoietic stem cell transplantation (HCT) from HLA-matched related donor for Fanconi anemia (FA) in adolescents and adults: a retrospective study by Eastern Mediterranean Blood and Marrow Transplantation Group (EMBMT)</title><source>SpringerLink Journals</source><source>Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals</source><creator>AlDawsari, Ghuzayel ; Elhaddad, Alaa ; El Fakih, Riad ; Ben Othman, Tarek ; Ahmed, Parvez ; Ghavamzadeh, Ardeshir ; Bazarbachi, Ali ; Dasouki, Majed J. ; Fathy, Gamal ; Alzahrani, Hazzaa ; Samra, Mohamed ; Torjemane, Lamia ; Satti, Tariq Mahmood ; Shaheen, Marwan ; Alfraih, Feras ; Ayas, Mouhab ; Alahmari, Ali ; Alhayli, Saud ; Nassar, Amr ; Abboud, Miguel ; Abdelfattah, Raafat ; El Solh, Hassan ; Hashmi, Shahrukh ; Elhassan, Tusneem ; Ahmed, Syed Osman ; Aljurf, Mahmoud</creator><creatorcontrib>AlDawsari, Ghuzayel ; Elhaddad, Alaa ; El Fakih, Riad ; Ben Othman, Tarek ; Ahmed, Parvez ; Ghavamzadeh, Ardeshir ; Bazarbachi, Ali ; Dasouki, Majed J. ; Fathy, Gamal ; Alzahrani, Hazzaa ; Samra, Mohamed ; Torjemane, Lamia ; Satti, Tariq Mahmood ; Shaheen, Marwan ; Alfraih, Feras ; Ayas, Mouhab ; Alahmari, Ali ; Alhayli, Saud ; Nassar, Amr ; Abboud, Miguel ; Abdelfattah, Raafat ; El Solh, Hassan ; Hashmi, Shahrukh ; Elhassan, Tusneem ; Ahmed, Syed Osman ; Aljurf, Mahmoud</creatorcontrib><description>Hematopoietic Stem Cell Transplantation (HSCT) is the only potentially curative treatment option for the hematologic complications that occur in patients with Fanconi anemia (FA). In this study, we present a retrospective multicenter analysis from the Eastern Mediterranean Blood and Marrow Transplantation Group (EMBMT) of matched related donor HSCT for FA in adolescents and adults transplanted between 1988 and 2015. Forty-five patients received HSCT with a median age at transplant of 18 years, the interquartile range (IQR) (15–23.5); 25 (55.6%) patients were females and 20 (44.4%) were males. Conditioning regimen was fludarabine-based in 29 (64.4%) patients, irradiation-based in five (11.1%) patients, and the remaining patients received other combinations. Indication for HSCT was bone marrow failure in 39 (86.7%) and myelodysplastic syndrome in six (13.3%) patients. Stem cell source was bone marrow in 22 (48.9%), peripheral blood in 20 (44.4%), umbilical cord blood in one (2.2%), and combination of bone marrow and cord blood in two (4.4%) patients. Twenty-seven (60%) patients engrafted and five (11.1%) had primary engraftment failure. The median time to neutrophil engraftment was 14 days (range 10–21 days); median time for platelet engraftment was 17 days (10–33 days). The probability of developing grade II–IV acute GVHD for all patients was 7.0% and chronic GVHD 36.6%. No new malignancies were reported. The OS probability was 53.6% (95% CI, 38.3–68.9%) with a median follow-up of 13 months (95% CI, 1–240). Our HLA-matched related HSCT results in AYA patients with FA compare favorably with other reported international registry data.</description><identifier>ISSN: 0268-3369</identifier><identifier>EISSN: 1476-5365</identifier><identifier>DOI: 10.1038/s41409-020-0809-5</identifier><identifier>PMID: 32024988</identifier><language>eng</language><publisher>London: Nature Publishing Group UK</publisher><subject>631/532/1542 ; 692/699/1541/13 ; Adolescents ; Adults ; Analysis ; Anemia ; Blood ; Bone marrow ; Care and treatment ; Cell Biology ; Complications ; Cord blood ; Correspondence ; Fanconi syndrome ; Fanconi's anemia ; Fludarabine ; Graft-versus-host reaction ; Hematology ; Hematopoietic stem cells ; Histocompatibility antigen HLA ; Internal Medicine ; Irradiation ; Medicine ; Medicine & Public Health ; Myelodysplastic syndrome ; Peripheral blood ; Public Health ; Radiation ; Stem cell transplantation ; Stem Cells ; Teenagers ; Transplantation ; Transplants & implants ; Umbilical cord ; Youth</subject><ispartof>Bone marrow transplantation (Basingstoke), 2020-07, Vol.55 (7), p.1485-1490</ispartof><rights>Springer Nature Limited 2020</rights><rights>COPYRIGHT 2020 Nature Publishing Group</rights><rights>Springer Nature Limited 2020.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c474t-669ec9198f5828c7ab494e27b70bf74b38e401e70170a053ddaeda3ab05683953</citedby><cites>FETCH-LOGICAL-c474t-669ec9198f5828c7ab494e27b70bf74b38e401e70170a053ddaeda3ab05683953</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://link.springer.com/content/pdf/10.1038/s41409-020-0809-5$$EPDF$$P50$$Gspringer$$H</linktopdf><linktohtml>$$Uhttps://link.springer.com/10.1038/s41409-020-0809-5$$EHTML$$P50$$Gspringer$$H</linktohtml><link.rule.ids>314,776,780,27901,27902,41464,42533,51294</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/32024988$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>AlDawsari, Ghuzayel</creatorcontrib><creatorcontrib>Elhaddad, Alaa</creatorcontrib><creatorcontrib>El Fakih, Riad</creatorcontrib><creatorcontrib>Ben Othman, Tarek</creatorcontrib><creatorcontrib>Ahmed, Parvez</creatorcontrib><creatorcontrib>Ghavamzadeh, Ardeshir</creatorcontrib><creatorcontrib>Bazarbachi, Ali</creatorcontrib><creatorcontrib>Dasouki, Majed J.</creatorcontrib><creatorcontrib>Fathy, Gamal</creatorcontrib><creatorcontrib>Alzahrani, Hazzaa</creatorcontrib><creatorcontrib>Samra, Mohamed</creatorcontrib><creatorcontrib>Torjemane, Lamia</creatorcontrib><creatorcontrib>Satti, Tariq Mahmood</creatorcontrib><creatorcontrib>Shaheen, Marwan</creatorcontrib><creatorcontrib>Alfraih, Feras</creatorcontrib><creatorcontrib>Ayas, Mouhab</creatorcontrib><creatorcontrib>Alahmari, Ali</creatorcontrib><creatorcontrib>Alhayli, Saud</creatorcontrib><creatorcontrib>Nassar, Amr</creatorcontrib><creatorcontrib>Abboud, Miguel</creatorcontrib><creatorcontrib>Abdelfattah, Raafat</creatorcontrib><creatorcontrib>El Solh, Hassan</creatorcontrib><creatorcontrib>Hashmi, Shahrukh</creatorcontrib><creatorcontrib>Elhassan, Tusneem</creatorcontrib><creatorcontrib>Ahmed, Syed Osman</creatorcontrib><creatorcontrib>Aljurf, Mahmoud</creatorcontrib><title>Outcome of hematopoietic stem cell transplantation (HCT) from HLA-matched related donor for Fanconi anemia (FA) in adolescents and adults: a retrospective study by Eastern Mediterranean Blood and Marrow Transplantation Group (EMBMT)</title><title>Bone marrow transplantation (Basingstoke)</title><addtitle>Bone Marrow Transplant</addtitle><addtitle>Bone Marrow Transplant</addtitle><description>Hematopoietic Stem Cell Transplantation (HSCT) is the only potentially curative treatment option for the hematologic complications that occur in patients with Fanconi anemia (FA). In this study, we present a retrospective multicenter analysis from the Eastern Mediterranean Blood and Marrow Transplantation Group (EMBMT) of matched related donor HSCT for FA in adolescents and adults transplanted between 1988 and 2015. Forty-five patients received HSCT with a median age at transplant of 18 years, the interquartile range (IQR) (15–23.5); 25 (55.6%) patients were females and 20 (44.4%) were males. Conditioning regimen was fludarabine-based in 29 (64.4%) patients, irradiation-based in five (11.1%) patients, and the remaining patients received other combinations. Indication for HSCT was bone marrow failure in 39 (86.7%) and myelodysplastic syndrome in six (13.3%) patients. Stem cell source was bone marrow in 22 (48.9%), peripheral blood in 20 (44.4%), umbilical cord blood in one (2.2%), and combination of bone marrow and cord blood in two (4.4%) patients. Twenty-seven (60%) patients engrafted and five (11.1%) had primary engraftment failure. The median time to neutrophil engraftment was 14 days (range 10–21 days); median time for platelet engraftment was 17 days (10–33 days). The probability of developing grade II–IV acute GVHD for all patients was 7.0% and chronic GVHD 36.6%. No new malignancies were reported. The OS probability was 53.6% (95% CI, 38.3–68.9%) with a median follow-up of 13 months (95% CI, 1–240). Our HLA-matched related HSCT results in AYA patients with FA compare favorably with other reported international registry data.</description><subject>631/532/1542</subject><subject>692/699/1541/13</subject><subject>Adolescents</subject><subject>Adults</subject><subject>Analysis</subject><subject>Anemia</subject><subject>Blood</subject><subject>Bone marrow</subject><subject>Care and treatment</subject><subject>Cell Biology</subject><subject>Complications</subject><subject>Cord blood</subject><subject>Correspondence</subject><subject>Fanconi syndrome</subject><subject>Fanconi's anemia</subject><subject>Fludarabine</subject><subject>Graft-versus-host reaction</subject><subject>Hematology</subject><subject>Hematopoietic stem cells</subject><subject>Histocompatibility antigen HLA</subject><subject>Internal Medicine</subject><subject>Irradiation</subject><subject>Medicine</subject><subject>Medicine & Public Health</subject><subject>Myelodysplastic syndrome</subject><subject>Peripheral blood</subject><subject>Public Health</subject><subject>Radiation</subject><subject>Stem cell transplantation</subject><subject>Stem Cells</subject><subject>Teenagers</subject><subject>Transplantation</subject><subject>Transplants & implants</subject><subject>Umbilical cord</subject><subject>Youth</subject><issn>0268-3369</issn><issn>1476-5365</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2020</creationdate><recordtype>article</recordtype><sourceid>BENPR</sourceid><recordid>eNp9ks2O0zAUhSMEYsrAA7BBlpBQuwj4N3Fm16naKVKr2ZS15SQ304wcO9gOaN6Yx8ClA0gjwcK6tu53j46vTpa9JfgjwUx-CpxwXOWY4hzLdBHPshnhZZELVojn2QzTQuaMFdVF9iqEe4wJ51i8zC4YxZRXUs6yH7dTbNwAyHXoCIOObnQ9xL5BIcKAGjAGRa9tGI22UcfeWTTfrg4L1Hk3oO1umaeh5ggt8mB0TLV11nnUpbPRtnG2R9rC0Gs03ywXqLdIt85AaMDGkFptek8mhiukk0T0LozQxP4bJAdT-4DqB7TWyYy3aA9tny7JDmiLro1z7S-BvfbefUeHJz5vvJtGNF_vr_eHxevsRadNgDeP9TL7slkfVtt8d3vzebXc5Q0vecyLooKmIpXshKSyKXXNKw60rEtcdyWvmQSOCZSYlFhjwdpWQ6uZrrEoJKsEu8zmZ93Ru68ThKiGPpzWmEy7KSjKBMWCCFwm9P0T9N5N3iZ3ivJSYE4EKf5PkSqJYSkT9eFM3WkD6gjaxGNwZjotIqhlQSUjRYITSM5gkzYdPHRq9P2g_YMiWJ1Spc6pUilV6pQqdfrTu0cLUz1A-2fid4wSQM9ASC17B_6vx3-r_gTOXdgf</recordid><startdate>20200701</startdate><enddate>20200701</enddate><creator>AlDawsari, Ghuzayel</creator><creator>Elhaddad, Alaa</creator><creator>El Fakih, Riad</creator><creator>Ben Othman, Tarek</creator><creator>Ahmed, Parvez</creator><creator>Ghavamzadeh, Ardeshir</creator><creator>Bazarbachi, Ali</creator><creator>Dasouki, Majed J.</creator><creator>Fathy, Gamal</creator><creator>Alzahrani, Hazzaa</creator><creator>Samra, Mohamed</creator><creator>Torjemane, Lamia</creator><creator>Satti, Tariq Mahmood</creator><creator>Shaheen, Marwan</creator><creator>Alfraih, Feras</creator><creator>Ayas, Mouhab</creator><creator>Alahmari, Ali</creator><creator>Alhayli, Saud</creator><creator>Nassar, Amr</creator><creator>Abboud, Miguel</creator><creator>Abdelfattah, Raafat</creator><creator>El Solh, Hassan</creator><creator>Hashmi, Shahrukh</creator><creator>Elhassan, Tusneem</creator><creator>Ahmed, Syed Osman</creator><creator>Aljurf, Mahmoud</creator><general>Nature Publishing Group UK</general><general>Nature Publishing Group</general><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>3V.</scope><scope>7QO</scope><scope>7QP</scope><scope>7T5</scope><scope>7U9</scope><scope>7X7</scope><scope>7XB</scope><scope>88E</scope><scope>8AO</scope><scope>8FD</scope><scope>8FE</scope><scope>8FH</scope><scope>8FI</scope><scope>8FJ</scope><scope>8FK</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>AZQEC</scope><scope>BBNVY</scope><scope>BENPR</scope><scope>BHPHI</scope><scope>CCPQU</scope><scope>DWQXO</scope><scope>FR3</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>GNUQQ</scope><scope>H94</scope><scope>HCIFZ</scope><scope>K9.</scope><scope>LK8</scope><scope>M0S</scope><scope>M1P</scope><scope>M7N</scope><scope>M7P</scope><scope>P64</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>PRINS</scope><scope>7X8</scope></search><sort><creationdate>20200701</creationdate><title>Outcome of hematopoietic stem cell transplantation (HCT) from HLA-matched related donor for Fanconi anemia (FA) in adolescents and adults: a retrospective study by Eastern Mediterranean Blood and Marrow Transplantation Group (EMBMT)</title><author>AlDawsari, Ghuzayel ; Elhaddad, Alaa ; El Fakih, Riad ; Ben Othman, Tarek ; Ahmed, Parvez ; Ghavamzadeh, Ardeshir ; Bazarbachi, Ali ; Dasouki, Majed J. ; Fathy, Gamal ; Alzahrani, Hazzaa ; Samra, Mohamed ; Torjemane, Lamia ; Satti, Tariq Mahmood ; Shaheen, Marwan ; Alfraih, Feras ; Ayas, Mouhab ; Alahmari, Ali ; Alhayli, Saud ; Nassar, Amr ; Abboud, Miguel ; Abdelfattah, Raafat ; El Solh, Hassan ; Hashmi, Shahrukh ; Elhassan, Tusneem ; Ahmed, Syed Osman ; Aljurf, Mahmoud</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c474t-669ec9198f5828c7ab494e27b70bf74b38e401e70170a053ddaeda3ab05683953</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2020</creationdate><topic>631/532/1542</topic><topic>692/699/1541/13</topic><topic>Adolescents</topic><topic>Adults</topic><topic>Analysis</topic><topic>Anemia</topic><topic>Blood</topic><topic>Bone marrow</topic><topic>Care and treatment</topic><topic>Cell Biology</topic><topic>Complications</topic><topic>Cord blood</topic><topic>Correspondence</topic><topic>Fanconi syndrome</topic><topic>Fanconi's anemia</topic><topic>Fludarabine</topic><topic>Graft-versus-host reaction</topic><topic>Hematology</topic><topic>Hematopoietic stem cells</topic><topic>Histocompatibility antigen HLA</topic><topic>Internal Medicine</topic><topic>Irradiation</topic><topic>Medicine</topic><topic>Medicine & Public Health</topic><topic>Myelodysplastic syndrome</topic><topic>Peripheral blood</topic><topic>Public Health</topic><topic>Radiation</topic><topic>Stem cell transplantation</topic><topic>Stem Cells</topic><topic>Teenagers</topic><topic>Transplantation</topic><topic>Transplants & implants</topic><topic>Umbilical cord</topic><topic>Youth</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>AlDawsari, Ghuzayel</creatorcontrib><creatorcontrib>Elhaddad, Alaa</creatorcontrib><creatorcontrib>El Fakih, Riad</creatorcontrib><creatorcontrib>Ben Othman, Tarek</creatorcontrib><creatorcontrib>Ahmed, Parvez</creatorcontrib><creatorcontrib>Ghavamzadeh, Ardeshir</creatorcontrib><creatorcontrib>Bazarbachi, Ali</creatorcontrib><creatorcontrib>Dasouki, Majed J.</creatorcontrib><creatorcontrib>Fathy, Gamal</creatorcontrib><creatorcontrib>Alzahrani, Hazzaa</creatorcontrib><creatorcontrib>Samra, Mohamed</creatorcontrib><creatorcontrib>Torjemane, Lamia</creatorcontrib><creatorcontrib>Satti, Tariq Mahmood</creatorcontrib><creatorcontrib>Shaheen, Marwan</creatorcontrib><creatorcontrib>Alfraih, Feras</creatorcontrib><creatorcontrib>Ayas, Mouhab</creatorcontrib><creatorcontrib>Alahmari, Ali</creatorcontrib><creatorcontrib>Alhayli, Saud</creatorcontrib><creatorcontrib>Nassar, Amr</creatorcontrib><creatorcontrib>Abboud, Miguel</creatorcontrib><creatorcontrib>Abdelfattah, Raafat</creatorcontrib><creatorcontrib>El Solh, Hassan</creatorcontrib><creatorcontrib>Hashmi, Shahrukh</creatorcontrib><creatorcontrib>Elhassan, Tusneem</creatorcontrib><creatorcontrib>Ahmed, Syed Osman</creatorcontrib><creatorcontrib>Aljurf, Mahmoud</creatorcontrib><collection>PubMed</collection><collection>CrossRef</collection><collection>ProQuest Central (Corporate)</collection><collection>Biotechnology Research Abstracts</collection><collection>Calcium & Calcified Tissue Abstracts</collection><collection>Immunology Abstracts</collection><collection>Virology and AIDS Abstracts</collection><collection>Health & Medical Collection</collection><collection>ProQuest Central (purchase pre-March 2016)</collection><collection>Medical Database (Alumni Edition)</collection><collection>ProQuest Pharma Collection</collection><collection>Technology Research Database</collection><collection>ProQuest SciTech Collection</collection><collection>ProQuest Natural Science Collection</collection><collection>Hospital Premium Collection</collection><collection>Hospital Premium Collection (Alumni Edition)</collection><collection>ProQuest Central (Alumni) (purchase pre-March 2016)</collection><collection>ProQuest Central (Alumni Edition)</collection><collection>ProQuest Central UK/Ireland</collection><collection>ProQuest Central Essentials</collection><collection>Biological Science Collection</collection><collection>ProQuest Central</collection><collection>Natural Science Collection</collection><collection>ProQuest One Community College</collection><collection>ProQuest Central Korea</collection><collection>Engineering Research Database</collection><collection>Health Research Premium Collection</collection><collection>Health Research Premium Collection (Alumni)</collection><collection>ProQuest Central Student</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>SciTech Premium Collection</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>ProQuest Biological Science Collection</collection><collection>Health & Medical Collection (Alumni Edition)</collection><collection>Medical Database</collection><collection>Algology Mycology and Protozoology Abstracts (Microbiology C)</collection><collection>Biological Science Database</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>ProQuest One Academic Eastern Edition (DO NOT USE)</collection><collection>ProQuest One Academic</collection><collection>ProQuest One Academic UKI Edition</collection><collection>ProQuest Central China</collection><collection>MEDLINE - Academic</collection><jtitle>Bone marrow transplantation (Basingstoke)</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>AlDawsari, Ghuzayel</au><au>Elhaddad, Alaa</au><au>El Fakih, Riad</au><au>Ben Othman, Tarek</au><au>Ahmed, Parvez</au><au>Ghavamzadeh, Ardeshir</au><au>Bazarbachi, Ali</au><au>Dasouki, Majed J.</au><au>Fathy, Gamal</au><au>Alzahrani, Hazzaa</au><au>Samra, Mohamed</au><au>Torjemane, Lamia</au><au>Satti, Tariq Mahmood</au><au>Shaheen, Marwan</au><au>Alfraih, Feras</au><au>Ayas, Mouhab</au><au>Alahmari, Ali</au><au>Alhayli, Saud</au><au>Nassar, Amr</au><au>Abboud, Miguel</au><au>Abdelfattah, Raafat</au><au>El Solh, Hassan</au><au>Hashmi, Shahrukh</au><au>Elhassan, Tusneem</au><au>Ahmed, Syed Osman</au><au>Aljurf, Mahmoud</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Outcome of hematopoietic stem cell transplantation (HCT) from HLA-matched related donor for Fanconi anemia (FA) in adolescents and adults: a retrospective study by Eastern Mediterranean Blood and Marrow Transplantation Group (EMBMT)</atitle><jtitle>Bone marrow transplantation (Basingstoke)</jtitle><stitle>Bone Marrow Transplant</stitle><addtitle>Bone Marrow Transplant</addtitle><date>2020-07-01</date><risdate>2020</risdate><volume>55</volume><issue>7</issue><spage>1485</spage><epage>1490</epage><pages>1485-1490</pages><issn>0268-3369</issn><eissn>1476-5365</eissn><abstract>Hematopoietic Stem Cell Transplantation (HSCT) is the only potentially curative treatment option for the hematologic complications that occur in patients with Fanconi anemia (FA). In this study, we present a retrospective multicenter analysis from the Eastern Mediterranean Blood and Marrow Transplantation Group (EMBMT) of matched related donor HSCT for FA in adolescents and adults transplanted between 1988 and 2015. Forty-five patients received HSCT with a median age at transplant of 18 years, the interquartile range (IQR) (15–23.5); 25 (55.6%) patients were females and 20 (44.4%) were males. Conditioning regimen was fludarabine-based in 29 (64.4%) patients, irradiation-based in five (11.1%) patients, and the remaining patients received other combinations. Indication for HSCT was bone marrow failure in 39 (86.7%) and myelodysplastic syndrome in six (13.3%) patients. Stem cell source was bone marrow in 22 (48.9%), peripheral blood in 20 (44.4%), umbilical cord blood in one (2.2%), and combination of bone marrow and cord blood in two (4.4%) patients. Twenty-seven (60%) patients engrafted and five (11.1%) had primary engraftment failure. The median time to neutrophil engraftment was 14 days (range 10–21 days); median time for platelet engraftment was 17 days (10–33 days). The probability of developing grade II–IV acute GVHD for all patients was 7.0% and chronic GVHD 36.6%. No new malignancies were reported. The OS probability was 53.6% (95% CI, 38.3–68.9%) with a median follow-up of 13 months (95% CI, 1–240). Our HLA-matched related HSCT results in AYA patients with FA compare favorably with other reported international registry data.</abstract><cop>London</cop><pub>Nature Publishing Group UK</pub><pmid>32024988</pmid><doi>10.1038/s41409-020-0809-5</doi><tpages>6</tpages><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0268-3369 |
| ispartof | Bone marrow transplantation (Basingstoke), 2020-07, Vol.55 (7), p.1485-1490 |
| issn | 0268-3369 1476-5365 |
| language | eng |
| recordid | cdi_proquest_miscellaneous_2352051507 |
| source | SpringerLink Journals; Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals |
| subjects | 631/532/1542 692/699/1541/13 Adolescents Adults Analysis Anemia Blood Bone marrow Care and treatment Cell Biology Complications Cord blood Correspondence Fanconi syndrome Fanconi's anemia Fludarabine Graft-versus-host reaction Hematology Hematopoietic stem cells Histocompatibility antigen HLA Internal Medicine Irradiation Medicine Medicine & Public Health Myelodysplastic syndrome Peripheral blood Public Health Radiation Stem cell transplantation Stem Cells Teenagers Transplantation Transplants & implants Umbilical cord Youth |
| title | Outcome of hematopoietic stem cell transplantation (HCT) from HLA-matched related donor for Fanconi anemia (FA) in adolescents and adults: a retrospective study by Eastern Mediterranean Blood and Marrow Transplantation Group (EMBMT) |
| url | https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2025-02-03T09%3A38%3A46IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-gale_proqu&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.genre=article&rft.atitle=Outcome%20of%20hematopoietic%20stem%20cell%20transplantation%20(HCT)%20from%20HLA-matched%20related%20donor%20for%20Fanconi%20anemia%20(FA)%20in%20adolescents%20and%20adults:%20a%20retrospective%20study%20by%20Eastern%20Mediterranean%20Blood%20and%20Marrow%20Transplantation%20Group%20(EMBMT)&rft.jtitle=Bone%20marrow%20transplantation%20(Basingstoke)&rft.au=AlDawsari,%20Ghuzayel&rft.date=2020-07-01&rft.volume=55&rft.issue=7&rft.spage=1485&rft.epage=1490&rft.pages=1485-1490&rft.issn=0268-3369&rft.eissn=1476-5365&rft_id=info:doi/10.1038/s41409-020-0809-5&rft_dat=%3Cgale_proqu%3EA628316920%3C/gale_proqu%3E%3Curl%3E%3C/url%3E&disable_directlink=true&sfx.directlink=off&sfx.report_link=0&rft_id=info:oai/&rft_pqid=2419205088&rft_id=info:pmid/32024988&rft_galeid=A628316920&rfr_iscdi=true |